Invus Financial Advisors

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Coave Therapeutics specializes in developing gene therapies for rare ocular and central nervous system (CNS) diseases. Their primary focus is creating treatments for rare retinal disorders using a recombinant adeno-associated virus vector, which delivers a functional copy of the PDE6B gene directly between photoreceptors and the retinal pigment epithelium to provide effective visual restoration.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS diseases.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative gene therapies utilizing its proprietary Anellovector platform. This platform, based on the human commensal virome, addresses several limitations of existing gene therapies, including restricted access to diverse tissues, challenges in redosing, risks of genomic integration, and issues with tolerability. Founded in 2017 and previously known as VL46, Inc., Ring Therapeutics aims to expand the applications of gene therapy beyond traditional gene replacement. By enabling a broader range of therapeutic modalities and mechanisms, the company seeks to treat a variety of previously inaccessible diseases, including genetic disorders, ophthalmology, oncology, and metabolic conditions.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.

LifeMine Therapeutics, Inc. is a biotechnology company focused on the genomic discovery of new drug modalities derived from eukaryotic microbes, specifically fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, it employs an integrated drug discovery platform that combines genomics, artificial intelligence, and synthetic biology. LifeMine’s Avatar-Rx platform utilizes high-throughput microbiology, data science, genome engineering, and automation technologies to identify novel gene-encoded molecules (GEMs) with specific biological functions. The company emphasizes chemoinformatic-assisted drug optimization and advanced chemical synthesis, enabling the progression of new product candidates into development. Founded by experts in the field, LifeMine Therapeutics aims to innovate medicine through its unique approach to leveraging the fungal biosphere.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative gene therapies utilizing its proprietary Anellovector platform. This platform, based on the human commensal virome, addresses several limitations of existing gene therapies, including restricted access to diverse tissues, challenges in redosing, risks of genomic integration, and issues with tolerability. Founded in 2017 and previously known as VL46, Inc., Ring Therapeutics aims to expand the applications of gene therapy beyond traditional gene replacement. By enabling a broader range of therapeutic modalities and mechanisms, the company seeks to treat a variety of previously inaccessible diseases, including genetic disorders, ophthalmology, oncology, and metabolic conditions.

Omega Therapeutics, Inc. is a development-stage biotechnology company based in Cambridge, Massachusetts, founded in 2016. The company specializes in developing genomic medicines aimed at curing diseases through precision tuning of the human genome. It utilizes its proprietary epigenomic programming platform to engineer a new class of programmable epigenetic medicines known as Omega Epigenomic Controllers. These controllers are designed to selectively regulate genomic activity, allowing for the downregulation or upregulation of genes to achieve therapeutic effects. Omega Therapeutics seeks to transform human medicine by harnessing the natural capacity of the genome to treat and cure various diseases.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Locanabio, Inc., based in San Diego, California, is a biotechnology company specializing in RNA-targeted gene therapies. Founded in 2016, the company focuses on developing treatments for severe neurodegenerative, neuromuscular, and retinal diseases. Locanabio's platform is designed to modify disease-causing RNA, offering therapeutic candidates distinct from traditional DNA-targeted approaches. The company aims to address a spectrum of underserved diseases, including those affecting the nervous system, muscles, and eyes.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing innovative therapeutic products for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts are dedicated to addressing disabling symptoms associated with conditions affecting the nervous system, such as Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma aims to enhance the quality of life and improve patient outcomes for those suffering from these challenging disorders.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on the discovery and development of gene therapies for retinal degenerative diseases and neurodegenerative disorders of the central nervous system. Founded in 2012, the company utilizes innovative technologies, including mitochondrial targeting sequences and optogenetics, to develop treatments that aim to preserve or restore vision in patients with severe vision impairments. Its lead product candidates include GS010, an AAV2-based gene therapy that is currently undergoing Phase III clinical trials for Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is in Phase I/II trials for retinitis pigmentosa and geographic atrophy associated with dry age-related macular degeneration. GenSight Biologics is also advancing other product candidates in preclinical development targeting various ophthalmic and neurodegenerative conditions.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS diseases.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Foghorn Therapeutics Inc. is a biopharmaceutical company focused on discovering and developing innovative medicines that target genetically determined dependencies within the chromatin regulatory system. Utilizing its proprietary Gene Traffic Control platform, Foghorn aims to manipulate gene expression to develop therapies for cancer and other serious diseases. The company is advancing multiple drug candidates, including FHD-286, a small-molecule enzymatic inhibitor designed for treating acute myeloid leukemia and uveal melanoma, and FHD-609, a protein degrader targeting BRD9 for synovial sarcoma. Additionally, Foghorn is engaged in preclinical and discovery programs that focus on selectively targeting BRM in non-small-cell lung cancer and developing modulators for ARID1B in bladder, ovarian, and endometrial cancers. Founded in 2015 and headquartered in Cambridge, Massachusetts, Foghorn Therapeutics collaborates with Merck Sharp & Dohme Corp. to explore novel oncology therapeutics.

KSQ Therapeutics, Inc., founded in 2015 and based in Cambridge, Massachusetts, specializes in precision functional genomics to discover innovative drug therapies. The company employs a novel approach to research and development that emphasizes de-risking prior to the creation of therapeutic candidates. Utilizing its proprietary CRISPRomics™ drug discovery engine, KSQ Therapeutics has mapped the functions of human genes across various diseases, enhancing its understanding of disease biology. This comprehensive insight enables the identification and validation of high-confidence, patient-specific drug targets. As a result, the company is focused on developing medicines that have significant potential to improve patient outcomes and has established a pipeline of preclinical discovery programs.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

Fluidigm Corporation develops and markets innovative technologies and tools for life sciences research globally. The company specializes in integrated fluidic circuits that control fluids at the nanoscale, enabling a range of applications in biological research. Its product offerings include analytical systems such as the Helios CyTOF system, the Hyperion imaging system, and the Biomark HD system, along with various assays and reagents designed for immuno-oncology and genetic analysis. Fluidigm also provides preparatory instruments and single-cell microfluidics, such as the C1 and Polaris systems. The company serves a diverse clientele, including academic institutions, clinical research laboratories, biopharmaceutical, biotechnology, and agricultural biotechnology companies, focusing on non-diagnostic and non-clinical applications. Established in 1999 as Mycometrix Corporation, it adopted the name Fluidigm Corporation in 2001 and is headquartered in South San Francisco, California.